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A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease
Bu çalışma, UX701 adlı bir gen terapisini, Wilson hastalığı (Wilson disease) olan yetişkinlerde standart tedaviyle (Standard of Care) karşılaştırarak inceliyor. Çalışmaya, ATP7B genindeki mutasyon ile tanısı doğrulanmış, en az iki aydır bakır bağlayıcı ve/veya çinko tedavisi altında stabil seyreden yetişkinler alınmaktadır. Çalışma Faz 1/Faz 2 aşamasında yürütülmüş; şu an katılımcı almamakta ancak devam etmektedir (active, not recruiting).
Yukarıdaki özet, resmî kaydın sade dile aktarılmış halidir. Orijinal İngilizce başlık referans için gösterilir.
Kriterler ClinicalTrials.gov kaydından orijinal İngilizce haliyle alınmıştır. Uygunluk kararını yalnızca deneyi yürüten ekip verebilir.
Key Inclusion Criteria: * Confirmed diagnosis of Wilson disease based on genetic confirmation of heterozygous or homozygous biallelic ATP7B mutation. * Stable Wilson disease as evidenced by ongoing copper chelator (ie, penicillamine, trientine) and/or zinc therapy for at least 2 months at screening, with no medication or dose changes for at least 2 months at screening. * Ongoing restriction of high copper containing foods for at least 2 months at Screening and continued through study participation. * Willing and able to comply with all study procedures and requirements, including frequent blood collection, total urine collection over a 24-hour period, patient-reported outcome assessments, and long-term follow-up Key Exclusion Criteria: * Detectable pre-existing antibodies to the AAV9 capsid. * Stage 1 only: History of copper chelator or zinc therapy noncompliance, in the Investigator's judgment, within 6 months prior to Screening. * History of liver transplant. * Active decompensated hepatic cirrhosis or history of hepatic encephalopathy. * Significant hepatic inflammation as evidenced by laboratory abnormalities. * Model for End-Stage Liver Disease (MELD) score \> 13. * Hemoglobin \< 9 g/dL * Presence of Stage 3 or higher chronic kidney disease based on estimated glomerular filtration rate \< 60 mL/min/1.73 m2. * Marked neurological deficit or compromise that, in the Investigator's opinion, would interfere with the subject's safety or ability to participate in the study. * Moderate to severe depression, recent or active suicidal ideation with intent or suicidal behavior, psychosis, or unstable psychiatric illness. * Known hypersensitivity to UX701 or its excipients, copper chelators, zinc, rituximab, tacrolimus, corticosteroids, or eculizumab that, in the Investigator's judgement, places the participant at increased risk for adverse events. * Participation in another gene transfer study or use of another gene transfer product before or during study participation. * Subjects with known hypersensitivity to amide-containing local anesthetics are excluded from participating in the optional liver biopsy substudy. Note: Other protocol defined Inclusion/ Exclusion criteria may apply